[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100604328":3},{"organization":4,"armGroups":7,"interventions":22,"overallOfficials":28,"centralContacts":29,"locations":35,"responsibleParty":120,"collaborators":28,"id":122,"slug":123,"hasResults":124,"nctId":125,"briefTitle":126,"officialTitle":127,"acronym":128,"eligibilityCriteria":129,"healthyVolunteers":124,"sex":130,"minAge":131,"maxAge":28,"enrollmentInfo":132,"targetDuration":28,"studyType":135,"phases":136,"briefSummary":138,"conditions":139,"keywords":141,"overallStatus":38,"whyStopped":28,"lastUpdateSubmitDate":147,"lastUpdatePostDateStruct":148,"startDateStruct":151,"completionDateStruct":153,"leadSponsor":155,"locationsCount":156},{"fullName":5,"class":6},"Solid Biosciences Inc.","INDUSTRY",[8,14,18],{"label":9,"type":10,"description":11,"interventionNames":12},"Cohort 1 (≥ 18 years of age)","EXPERIMENTAL","Participants will receive a single intravenous (IV) infusion (dose 1) of SGT-501.",[13],"Drug: SGT-501",{"label":15,"type":10,"description":16,"interventionNames":17},"Cohort 2 (≥ 18 years of age)","Participants will receive a single IV infusion (dose 2) of SGT-501.",[13],{"label":19,"type":10,"description":20,"interventionNames":21},"Cohort 3 (≥ 7 to \u003C 18 years)","Participants will receive a single IV infusion (level at or below dose(s) assessed in adults) of SGT-501.",[13],[23],{"type":24,"name":25,"description":26,"armGroupLabels":27,"otherNames":28},"DRUG","SGT-501","IV for infusion",[9,15,19],null,[30],{"name":31,"role":32,"phone":33,"phoneExt":28,"email":34},"Solid Bio Clinical Trials","CONTACT","617-337-4680","clinicaltrials@solidbio.com",[36,54,72,89,100],{"facility":37,"status":38,"city":39,"state":40,"zip":41,"country":42,"countryCode":43,"cosmosGeoPoint":44,"geoPoint":49,"contacts":50},"Boston Children's Hospital","RECRUITING","Boston","Massachusetts","02459","United States","US",{"type":45,"coordinates":46},"Point",[47,48],-71.05977,42.35843,{"lat":48,"lon":47},[51],{"name":52,"role":32,"phone":28,"phoneExt":28,"email":53},"Elizabeth DeWitt, MD","gene.therapy@childrens.harvard.edu",{"facility":55,"status":38,"city":56,"state":57,"zip":58,"country":42,"countryCode":43,"cosmosGeoPoint":59,"geoPoint":63,"contacts":64},"Mayo Clinic","Rochester","Minnesota","55905",{"type":45,"coordinates":60},[61,62],-92.4699,44.02163,{"lat":62,"lon":61},[65,69],{"name":66,"role":32,"phone":67,"phoneExt":28,"email":68},"Kolawole Olarinoye","507-293-1564","Olarinoye.Kolawole@mayo.edu",{"name":70,"role":71,"phone":28,"phoneExt":28,"email":28},"John Giudicessi, MD","PRINCIPAL_INVESTIGATOR",{"facility":73,"status":38,"city":74,"state":75,"zip":76,"country":42,"countryCode":43,"cosmosGeoPoint":77,"geoPoint":81,"contacts":82},"Cleveland Clinic","Cleveland","Ohio","44195",{"type":45,"coordinates":78},[79,80],-81.69541,41.4995,{"lat":80,"lon":79},[83,87],{"name":84,"role":32,"phone":85,"phoneExt":28,"email":86},"Paige Ewing","216-445-8798","ewingp@ccf.org",{"name":88,"role":71,"phone":28,"phoneExt":28,"email":28},"Peter Aziz, MD",{"facility":90,"status":91,"city":92,"state":93,"zip":94,"country":42,"countryCode":43,"cosmosGeoPoint":95,"geoPoint":99,"contacts":28},"University of Pennsylvania","NOT_YET_RECRUITING","Philadelphia","Pennsylvania","19104",{"type":45,"coordinates":96},[97,98],-75.16362,39.95238,{"lat":98,"lon":97},{"facility":101,"status":38,"city":102,"state":103,"zip":104,"country":105,"countryCode":106,"cosmosGeoPoint":107,"geoPoint":111,"contacts":112},"St. Paul's Hospital","Vancouver","British Columbia","BC V6Z 1Y6","Canada","CA",{"type":45,"coordinates":108},[109,110],-123.11934,49.24966,{"lat":110,"lon":109},[113,118],{"name":114,"role":32,"phone":115,"phoneExt":116,"email":117},"Brianna Davies","604-682-2344","64252","brianna.davies@phc.ca",{"name":119,"role":71,"phone":28,"phoneExt":28,"email":28},"Thomas Roston, MD",{"type":121,"investigatorFullName":28,"investigatorTitle":28,"investigatorAffiliation":28,"oldNameTitle":28,"oldOrganization":28},"SPONSOR","100604328","phase-1-a-study-of-sgt-501-gene-therapy-in-catecholaminergic-polymorphic-ventricular-tachycardia-cpvt-100604328",false,"NCT07148089","A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)","A Phase 1b, Multicenter, Open-Label, Dose Finding Study to Investigate the Safety and Tolerability of a Single Intravenous Dose of SGT-501 in Patients With Catecholaminergic Polymorphic Ventricular Tachycardia","ARTEMIS","Inclusion Criteria:\n\nType of Participant and Disease Characteristics:\n\n* Clinical diagnosis of CPVT, based on documented history of polymorphic or bidirectional non-sustained ventricular tachycardia with exercise or ventricular ectopy in a pattern consistent with CPVT on EST.\n* Central Screening laboratory determination of a RYR2 variant that is pathogenic or likely pathogenic for CPVT.\n* Documented history of life-threatening ventricular arrhythmic event defined as: survived sudden cardiac arrest, sudden cardiac arrest with appropriate implantable cardioverter defibrillator (ICD) shock, arrhythmic syncope, or sustained ventricular tachycardia (30 seconds or more) with or without ICD shock.\n* On stable dose (defined as no change in dose by more than 50% for at least 1 month prior to Screening) of standard-of-care therapy defined as a beta-blocker and\u002For flecainide.\n* Documented prior history of EST demonstrating a ventricular arrythmia score (VAS) score of ≥ 2.\n* For the first 2 participants in each cohort only: a properly functioning ICD device in place. Following review of data from Cohorts 1 and 2, the Data Safety and Monitoring Board (DSMB) will determine if this criterion is required for participants in Cohort 3.\n* Must be up to date with meningococcal vaccination per national guidelines or willing to receive meningococcal vaccine to achieve this.\n* Other inclusion criteria to be applied as per protocol.\n\nExclusion Criteria:\n\n* Abnormal liver function: gamma-glutamyl transferase (GGT) \\> 1.5 × upper limit of normal \\[ULN\\] or total bilirubin \\> ULN).\n* Abnormal renal function defined by estimated glomerular filtration rate \\\u003C 60 milliliter \u002Fminute (mL\u002Fmin)\u002F1.73-square meter (m\\^2) using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) Formula.\n* Clinically significant abnormalities of coagulation including international normalized ratio or activated partial thromboplastin time \\> 1.2 × ULN or platelets \\\u003C 150,000 cells\u002Fcubic millimeter (mm\\^3).\n* Potential concomitant cardiomyopathy or inherited arrhythmia as evidenced by pathogenic or likely pathogenic mutation other than RYR2 obtained on cardiac panel during Screening.\n* Current or prior treatment with an approved or investigational gene transfer drug.\n* Exposure to another investigational drug within 90 days prior to Screening or 5 half-lives since last administration, whichever is longer.\n* Contraindication or unwillingness to receive required immunosuppression regimen.\n* Body mass index ≥ 30 kilograms per square meter (kg\u002Fm\\^2).\n* Other exclusion criteria to be applied as per protocol.","ALL","7 Years",{"count":133,"type":134},18,"ESTIMATED","INTERVENTIONAL",[137],"PHASE1","This is a Phase 1b, Multicenter, Open-Label, Dose Finding Study to Investigate the Safety and Tolerability of a Single Intravenous Dose of SGT-501 in participants with catecholaminergic polymorphic ventricular tachycardia (CPVT). The first-in-human (FIH) safety study will focus on obtaining safety data in adult participants. Cohort 1 and Cohort 2 (optional for dose exploration) will include participants ≥ 18 years of age. Cohort 3 will include participants ≥ 7 to \\\u003C 18 years of age and will be initiated following data and safety monitoring board (DSMB) recommendations. Participants will be monitored for 5 years post-administration of SGT-501 including the active treatment period (1 year) and long-term follow-up (LTFU) (4 years) period.",[140],"Catecholaminergic Polymorphic Ventricular Tachycardia",[142,25,143,144,145,146],"Catecholaminergic polymorphic ventricular tachycardia (CPVT)","Ryanodine Receptor 2 (RYR2)","adeno-associated virus serotype 8 (AAV8)","Cardiac","Gene Therapy","2026-06-01",{"date":149,"type":150},"2026-06-02","ACTUAL",{"date":152,"type":150},"2026-02-23",{"date":154,"type":134},"2031-05",{"name":5,"class":6},5]